Apheresis Device Using HTT Binding Molecules to Lower Plasma Huntingtin
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Solution Overview
Problem
Current treatments for Huntington's disease focus on symptom management, with no cure or prevention available, and existing therapeutic approaches face challenges in delivering agents to affected neurons in the brain.
Innovation Solution
An apheresis device using HTT binding molecules, such as antibodies specifically targeting Huntingtin protein or fragments, to capture and remove Huntingtin from the blood plasma, thereby addressing the systemic nature of the disease.
Engineering Contradictions & Design Principles
Engineering Contradiction Analysis
1Reliability
If conventional therapeutic agents are used to target Huntingtin in the brain, then disease progression can be addressed, but delivery to affected neurons remains unsuccessful
Solution Approach 1:
The patent uses plasma as an intermediary medium to deliver HTT binding molecules to the brain. Instead of directly injecting agents into the brain or using complex neural delivery systems, the invention circulates binding molecules through the blood plasma, allowing them to cross the blood-brain barrier and reach affected neurons naturally, thus simplifying the delivery system while improving reliability
Solution Approach 2:
The invention replaces complex mechanical delivery systems (such as intracranial injections, viral vectors, or nanocarriers) with a biological mechanism - the natural circulation of blood plasma. This substitution eliminates the need for invasive mechanical procedures while achieving effective delivery of therapeutic molecules to the brain
2Reliability
If Huntingtin lowering strategies are implemented, then disease pathology can be addressed, but lack of delivery to targeting site prevents effectiveness
Solution Approach 1:
The invention leverages the body's own circulatory system to deliver the therapeutic agent. The HTT binding molecules are administered intravenously and automatically circulate through the blood plasma to the brain, utilizing the body's natural self-service delivery mechanism rather than requiring external mechanical assistance or complex administration procedures
Solution Approach 2:
The blood plasma serves multiple functions: it acts as the delivery vehicle for HTT binding molecules, provides a natural pathway across the blood-brain barrier, and enables continuous circulation to reach all affected neurons. This multi-functionality simplifies administration while ensuring reliable therapeutic delivery
Applied Scientific Principles
This section explains which scientific principles are used to turn an abstract innovation direction into a practical engineering solution.
Function Achieved in This Case
The apheresis device effectively lowers Huntingtin expression in primary human macrophages/monocytes, potentially reversing disturbances in the innate immune system and providing a therapeutic benefit by reducing plasma Huntingtin levels.
Implementation Method 1
An apheresis device using HTT binding molecules, such as antibodies specifically targeting Huntingtin protein or fragments, to capture and remove Huntingtin from the blood plasma
Implementation Method 2
An apheresis device using HTT binding molecules to capture and remove Huntingtin from the blood plasma
Data Source
AI summary
Disclosed is an apheresis device including a solid carrier capable of being contacted with the blood or plasma flow, characterised in that the solid carrier includes one or several HTT-binding molecule(s) capable of adsorbing HTT or fragments thereof in a specific manner from plasma or blood or other HTT containing body fluids such as CSF.